IACC Autism Spectrum Disorder Research Portfolio Analysis: Project Listing
Project Id Project Number Sub Project Number Funder Principal Investigator Project Title Project Description Fiscal Year Funding Category Strategic Plan Objective Objective Status Code Arra Funded? Federal/Private Institution State/Country Project Status Web Link 1 Web Link 2 Web Link 3 Active Current Award Period
Project Id: 3736 2368 None Autism Speaks Arnold, L. Eugene Neuronal nicotinic receptor modulation in the treatment of autism: A pilot trial of mecamylamine One consistently identified abnormality in autism is a reduction in one of the receptors (nicotinic) for the neurotransmitter acetylcholine. The proposed pilot trial aims to test the response of individuals with autism to agents that target this receptor type, thus changing acetylcholine signaling. There is one nicotinic agent that has been shown to be safe in other childhood-onset disorders. This agent will be pilot-tested in children with autism. Twenty children with autism spectrum disorder will receive the drug or placebo over 13 weeks in a controlled (placebo double-blind) trial. The outcome will be assessed by standard clinical rating scales which will measure a range of behavioral and communicative functions, including attention. Computerized testing will also be used to assess changes in attention objectively. $0.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Private The Ohio State University Ohio Ongoing http://www.autismspeaks.org/science/grants/neuronal-nicotonic-receptor-modulation-treatment-autism-pilot-trial-mecamylamine?destination=science%2Fgrant-search%2Fresults%2FNeuronal+nicotinic+receptor+modulation+in+the+treatment+of+autism%3A+A+pilot+trial+of+mecamylamine No URL available. No URL available. active 2007-2010
Project Id: 3743 5R21HD065276-02 None National Institutes of Health Bartz, Jennifer The effects of oxytocin on complex social cognition in autism spectrum disorders Social impairment is a central, unifying feature of autism and autism spectrum disorders (ASD), and perhaps also the most debilitating, but there has been little headway in the development of pharmacological treatments for the social domain. The purpose of this research is to determine whether oxytocin-a peptide hormone that plays a role in affiliative behavior in animals facilitates complex social cognition in adults with ASD and to elucidate the neurobiological mechanism involved in this process to clarify the viability of this potentially ground breaking treatment. Specifically, 35 adults with ASD will randomly receive a single dose of synthetic oxytocin, administered via nasal spray, or a matching placebo on two separate occasions. Researchers will then assess performance on a novel, complex social cognition task as well as brain activation using functional magnetic resonance imaging (fMRI). If positive, this research will point to a novel, potentially groundbreaking therapeutic candidate to target social dysfunction in ASD. Moreover, the focus on adults with ASD will be an important contribution to the field of autism because to date there is a paucity of effective interventions for adults with ASD. $285,221.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. Yes Federal Mount Sinai School of Medicine New York Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=7941044&icde=8942834 No URL available. No URL available. active 2009-2011
Project Id: 3820 5R01MH086784-02 None National Institutes of Health Casanova, Manuel Building a selective inhibitory control tone in autism: An rTMS study Some reports have shown that autism is associated with structural abnormalities in the brain. A GABAergic interneuron called a double-bouquet cell usually protects the areas around minicolumns, which help give the cortical area of the brain structure. Having too many axon terminals, dendrites, and other cell matter too close to the minicolums has been associated with autism. This study will try to strengthen double-bouquet cells to increase their power to inhibit encroachment on the space around the minicolumns. This will be done in autistic patients and controls by inducing electrical current into the double-bouquet cells (by creating a magnetic field) through Transcranial Magnetic Stimulation (TMS). Outcome measures will include behavioral and electrophysiological measures. The results of the proposed study will help understand the specific social communication and cognitive deficits associated with developmental abnormalities of functions within cortical circuitry, and thereby contribute to understanding the brain substrates of behavioral dysfunctions typical for autism. The research represents a new development in combining TMS with functional outcome measures (cognitive event-related potential (ERP), EEG), using TMS as a theory-guided psychiatric therapy in autism. $222,000.00 Treatments Question 4: Other Dot not applicable No Federal University of Louisville Kentucky Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=7874720&icde=6646667 No URL available. No URL available. active 2009-2013
Project Id: 3983 5U01NS061264-03 1 National Institutes of Health Chugani, Diane ACE Network: Early pharmacotherapy guided by biomarkers in autism This study is part of an Autism Centers of Excellence (ACE) Network. Previous research has shown that synthesis of the neurotransmitter serotonin is abnormal during critical periods of brain and synapse development in autistic children. The researchers hypothesize that one pharmacological approach to the treatment of autism is the use of serotonin agonists in children less than the age of six years. Following up on smaller pilot studies, this study is a randomized clinical trial of the serotonin agonist buspirone in a large group of young children with ASD. In this trial, researchers will test the safety and efficacy of buspirone in the treatment of core features of autism and associated behavioral dysfunction in young children with autism. Researchers will also employ a biomarker measure for serotonin synthesis capacity to assess patient differences in response to treatment. The researchers hypothesize that buspirone treatment effects on serotonin synthesis capacity will correlate with positive changes in social interaction, repetitive behavior, sensory dysfunction and anxiety. If buspirone is found to be safe and effective, it would represent a novel pharmacological treatment for core symptoms in ASDs. $1,000,000.00 Treatments Question 4: Short-term Objective F Green dot: Objective has greater than or equal to the recommended funding. No Federal Wayne State University Michigan Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=8053739&icde=8916724 No URL available. No URL available. active 2008-2013
Project Id: 3751 1R01MH086927-01A1 None National Institutes of Health King, Bryan 2/4-RUPP autism network: Guanfacine for the treatment of hyperactivity in PDD This is a multi-site collaborative research project from The Research Units on Pediatric Psychopharmacology [RUPP] Autism Network (Indiana University, Seattle Children's Research Institute, UCLA, and Yale University). Recent data indicate that as many as 50% of children with pervasive developmental disorders (PDDs) have moderate to severe problems of hyperactivity and impulsiveness. Consensus is lacking on how to treat children with PDD accompanied by hyperactivity. Compared to typically developing children with ADHD, children with PDD often show less benefit and greater side effect burden. Guanfacine is commonly used in this population, but poorly studied. Pilot data indicate that guanfacine is a promising treatment for hyperactivity in children with PDD with a good tolerability profile. In addition, biomarkers have been identified (genetic and neurochemical) that may be associated with positive effects. This study involves an 8-week randomized, double-blind, placebo- controlled trial of guanfacine for 170 children (ages 5-13 years) with PDD accompanied by hyperactivity and impulsiveness. Subjects who show a positive response will be invited to enter an 8-week Extension phase (treatment mask will not be broken). The treatment blind will be broken for children who do not achieve a positive response in the double-blind phase. Children who show no change or deterioration on placebo will be treated with guanfacine in an 8-week Open-label phase. Children who show a partial response to guanfacine will be randomly assigned to a 4-week add-on trial of methylphenidate or placebo to evaluate the potential benefits of combined treatment. Fifty subjects are expected to enter this pilot trial. The role of gene variants and urinary adrenergic/noradrenergic measures as biomarkers in moderating response to guanfacine on primary efficacy measures and adverse effects will also be explored. $325,136.00 Treatments Question 4: Long-term Objective C Green dot: Objective has greater than or equal to the recommended funding. No Federal Seattle Children's Hospital Washington New http://projectreporter.nih.gov/project_info_description.cfm?aid=7889283&icde=8916618 No URL available. No URL available. active 2010-2013
Project Id: 3755 1R01MH083707-01A2 None National Institutes of Health Scahill, Lawrence 4/4-RUPP Autism Network: Guanfacine for the treatment of hyperactivity in PDD This is a multi-site collaborative research project from The Research Units on Pediatric Psychopharmacology [RUPP] Autism Network (Indiana University, Seattle Children's Research Institute, UCLA, and Yale University). Recent data indicate that as many as 50% of children with pervasive developmental disorders (PDDs) have moderate to severe problems of hyperactivity and impulsiveness. Consensus is lacking on how to treat children with PDD accompanied by hyperactivity. Compared to typically developing children with ADHD, children with PDD often show less benefit and greater side effect burden. Guanfacine is commonly used in this population, but poorly studied. Pilot data indicate that guanfacine is a promising treatment for hyperactivity in children with PDD with a good tolerability profile. In addition, biomarkers have been identified (genetic and neurochemical) that may be associated with positive effects. This study involves an 8-week randomized, double-blind, placebo- controlled trial of guanfacine for 170 children (ages 5-13 years) with PDD accompanied by hyperactivity and impulsiveness. Subjects who show a positive response will be invited to enter an 8-week Extension phase (treatment mask will not be broken). The treatment blind will be broken for children who do not achieve a positive response in the double-blind phase. Children who show no change or deterioration on placebo will be treated with guanfacine in an 8-week Open-label phase. Children who show a partial response to guanfacine will be randomly assigned to a 4-week add-on trial of methylphenidate or placebo to evaluate the potential benefits of combined treatment. Fifty subjects are expected to enter this pilot trial. The role of gene variants and urinary adrenergic/noradrenergic measures as biomarkers in moderating response to guanfacine on primary efficacy measures and adverse effects will also be explored. $564,924.00 Treatments Question 4: Long-term Objective C Green dot: Objective has greater than or equal to the recommended funding. No Federal Yale University Connecticut New http://projectreporter.nih.gov/project_info_description.cfm?aid=7887234&icde=8916019 No URL available. No URL available. active 2010-2013
Project Id: 3982 7R01MH079080-04 None National Institutes of Health Aman, Michael 2/3-Atomoxetine placebo and parent training in autism There have been relatively few randomized clinical trials of pharmacology for attention-deficit/hyperactivity (ADHD) in children with autism spectrum disorders (ASD). Atomoxetine (ATX) was approved by the FDA in January 2003 for treatment of ADHD, but only 4 studies of this drug have been conducted in children with ASD. Behavioral treatments, such as training parents in the use of behavioral interventions (PMT), are also standard treatments for ADHD in typically developing children. While PMT also has long been used in ASD, it has rarely been studied in combination with psychopharmacologic treatment in this population. This three-pronged, 10-week trial in children with ASD will compare atomoxetine vs. placebo, PMT vs. no-PMT, and combined ATX/PMT vs. PMT alone. This trial has the potential to provide answers regarding optimal treatment (both psychosocial and pharmacological) for a sizeable subgroup of children with ASD who experience symptoms of over activity and inattention. $361,873.00 Treatments Question 4: Short-term Objective F Green dot: Objective has greater than or equal to the recommended funding. No Federal Ohio State University Ohio Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=8046736&icde=6645200 No URL available. No URL available. active 2008-2013
Project Id: 3738 5735 None Autism Speaks Sahin, Mustafa Randomized phase 2 trial of RAD001 (an MTOR inhibitor) in patients with tuberous sclerosis complex Autism spectrum disorder (ASD) is a major public health problem that disrupts families and leads to significant disability. There is no treatment aimed at the neurobiological bases of ASD. Tuberous sclerosis complex (TSC) provides an ideal model for autism research, given the very high rates of autism spectrum disorders associated with TSC and its increasingly well-characterized genetic and molecular basis. Breakthrough findings are emerging from studies of both animal models and children with TSC, of which 25-60% have ASD. The undisputed identification of two genes that cause TSC has led to the development of mouse models for TSC, which allows researchers to study the particular deleterious effects of the genetic mutations on brain functioning. There is promising evidence that these genes may cause defects in brain wiring and the connections among brain cells, and that these wiring abnormalities can be prevented or reversed by a class of drugs: the mTOR inhibitors. This Phase II randomized, placebo-controlled trial study will evaluate the efficacy of RAD001, an mTOR inhibitor, in 6-21 year old individuals with TSC, to test the hypothesis that mTOR inhibition may improve aspects of neurocognition in children and young adults with TSC. It will use a broad battery of neurocognitive tests to evaluate the study participants before, during and after treatment. While this trial is limited to individuals with TSC, the mTOR pathway has been implicated in other ASD-associated diseases such as Fragile X and patients with PTEN mutations. Therefore, it is hoped that findings from this study, although limited to individuals with TSC, may have implications for other subpopulations of individuals with ASD in the future. $65,000.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Private Childrens Hospital Boston Massachusetts New http://www.autismspeaks.org/science/grants/randomized-phase-2-trial-rad001-mtor-inhibitor-patients-tuberous-sclerosis-complex?destination=science%2Fgrant-search%2Fresults%2FSahin%2C+Mustafa No URL available. No URL available. active 2010-2013
Project Id: 3992 5R01MH083247-03 None National Institutes of Health Smith, Tristram 3/3-Atomoxetine placebo and parent training in autism While a sizeable subgroup of children with ASD also experience symptoms of over activity and inattention, there have been relatively few randomized clinical trials of pharmacology, especially in combination with behavioral therapy, for attention-deficit/hyperactivity (ADHD) in children with autism spectrum disorders (ASD). Studies involving stimulant medications have generally found poorer response rates and higher rates of intolerable side effects than in typically developing children with ADHD. Atomoxetine (Strattera) – a nonstimulant that affects the norepineprine system – was approved by the FDA in January 2003 for treatment of ADHD, but the relative effectiveness of atomoxetine in ASD and in combination with evidence-based behavioral interventions has not been well examined. In this project, researchers will perform a double-blind, placebo-controlled, 10-week parallel-group comparison of atomoxetine (ATX) versus placebo in ASD children with ADHD. In a separate but parallel study, the researchers will also assess the effectiveness of combining ATX treatment with a standard behavioral intervention paradigm known as Parent Management Training (PMT), which alone has been shown to be useful in managing ADHD symptoms. Children with ASD have been neglected in pharmacological research, placing an enormous burden on health care and educational systems. This trial has the potential to provide answers regarding optimal treatment (both behavioral and pharmacological) for a sizeable subgroup of children with ASD who experience symptoms of over activity and inattention. $277,198.00 Treatments Question 4: Short-term Objective F Green dot: Objective has greater than or equal to the recommended funding. No Federal University of Rochester New York Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=7799934&icde=6645241 No URL available. No URL available. active 2008-2013
Project Id: 3739 n/a None Autism Speaks Staff Member Clinical Trials Network The Autism Clinical Trials Network (ACTN) is a collaboration of treatment and research centers dedicated to accelerating clinical trials of investigational treatments for autism and to increasing the number of biological treatments available to families and clinicians. The ACTN was officially launched in 2005 to focus on translational research, the critical step necessary to transfer findings from the laboratory to clinical application. By facilitating data-sharing and multi-center trials, the goal of the network will be to test the efficacies of treatment approaches as well as to rapidly screen new promising compounds that emerge from biomedical research, significantly decreasing the time and amount of money needed to develop them. $0.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Private Autism Speaks (AS) New York Ongoing http://www.autismspeaks.org/science/resources-programs/autism-clinical-trials-network No URL available. No URL available. active Ongoing
Project Id: 3742 AS073084P1 None Department of Defense Stein, Peter Developing treatment, treatment validation, and treatment scope in the setting of an autism clinical trial This proposal has three linked and interacting projects designed to (1) develop new therapy for children with autism, (2) develop means of validating and monitoring responses to therapy assessed through clinical changes in the children and the amount of certain lipid biomarkers that they excrete in their urine, and (3) develop means for selecting children with autism likely to respond to this therapy based upon the children's genotypes, that is, the pattern of variants of certain key genes that the children have.The rationale is that growing evidence supports oxidative stress as a mechanism that contributes to autism. A key omega-3 fatty acid called docosahexaenoic acid (DHA) is a normal substance that is present in large amounts in the brain and that has properties that oppose oxidative stress. Therapeutic trials of agents containing DHA appear promising in autism. We were first to find that urinary excretion of a lipid biomarker of oxidative stress is increased in autism. In a preliminary study, we found that the amount of this biomarker excreted in children with autism correlated with a certain genotype in the children.We propose to carry out a rigorous therapeutic trial to assess the effect of DHA vs. placebo treatment on the global severity, behavioral symptoms, and functional ability of child and adolescent autistic disorder, in a 12-week double blind placebo-controlled parallel study (Project 1). We propose to measure changes in urinary excretion of this biomarker, isoprostane, during DHA therapy in the children with autism and develop new urinary biomarkers that may be even more informative (Project 2). We also propose to determine genotypes of the children undergoing DHA therapy since we found one gene variant, GSTM1*0, that correlated with the amount of isoprostane that children with autism excreted in their urine in a preliminary study (Project 3). We will test whether this gene variant and new gene variants that we will study will correlate also with response to DHA treatment (Project 3). We hope that this will allow us to predict which children with autism will respond to DHA therapy. This may also give us insight into whether some children with autism have a gene variant that interferes with DHA metabolism.This will help all children with autism by developing a new method of therapy, by developing a method to monitor the success of the therapy and show that it is working and by developing a method to determine which children with autism will respond best to the therapy.The clinical benefits will be assessed for global severity of autism, and for behavioral and functional aspects of autism. The therapy is safe and has no known risks.What is the projected time to achieve a consumer-related outcome? The study is a direct therapeutic trial. If the trial is successful, this therapy will be available to consumers by the end of the three-year project.The likely contributions of this study are (1) development of new therapy for autism, (2) development of a way to determine if the therapy is working in a child with autism and how well it is working, and (3) to develop a way of determining which children will respond best to the therapy. $0.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal University of Medicine & Dentistry of New Jersey New Jersey Ongoing http://cdmrp.army.mil/search.aspx No URL available. No URL available. active 2008-2012
Project Id: 3756 5K23MH082119-03 None National Institutes of Health Stigler, Kimberly Pharmacotherapy of pervasive developmental disorders This is a mentored Research Career Development Award to support the career development of clinical investigators in patient-oriented research. Pervasive Developmental Disorder-Not Otherwise Specified (PDD-NOS) is a highly prevalent disorder often associated with severe irritability that impedes participation in educational, therapeutic, and vocational programs, with escalating costs of care. Thus, the development of safe and effective pharmacotherapies that improve irritability is clearly relevant to public health. The investigator will conduct a double-blind, placebo-controlled and open-label continuation study of aripiprazole for irritability in 60 children and adolescents with PDD-NOS. $184,587.00 Treatments Question 4: Long-term Objective C Green dot: Objective has greater than or equal to the recommended funding. No Federal Indiana University-Purdue University Indianapolis Indiana Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=7888140&icde=8965006 No URL available. No URL available. active 2008-2013
Project Id: 3746 1ZIAMH002913-03 None National Institutes of Health Swedo, Susan Trial of a glutamate antagonist in the treatment of OCD and autistic disorders Riluzole, a glutamate antagonist, reduces symptoms in adults with obsessive-compulsive disorder (OCD). Preliminary data will be collected on Riluzole efficacy and safety in children and adolescents with co-morbid conditions of ASD and OCD. $770,674.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal National Institutes of Health Maryland Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=8158152&icde=8917101 No URL available. No URL available. active Ongoing
Project Id: 3981 R40 MC 19926 None Health Resources and Services Administration Beversdorf, David Predictors of effects of propranolol on language & connectivity in autism This project examines how markers of sympathetic reactivity predict response to propranolol in autism. $184,288.00 Treatments Question 4: Short-term Objective F Green dot: Objective has greater than or equal to the recommended funding. No Federal University of Missouri Missouri New http://www.mchb.hrsa.gov/research/project_info.asp?ID=153 No URL available. No URL available. active 2010-2012
Project Id: 3984 1R01HD062550-01A1 None National Institutes of Health Devane, C. Lindsay Biomarkers in Autism of Aripiprazole and Risperidone Treatment (BAART) The Biomarkers in autism of aripiprazole and risperidone treatment (BAART) project will provide evidence-based guidance in the selection and monitoring of drug treatment of autism. BAART involves three academic centers across South Carolina, with expertise in phenotyping patients with autistic spectrum disorders, assessing patient response in clinical trials, and expertise in pharmacogenomic research. Although the FDA has approved use of the antipsychotic drug risperidone for irritability associated with autistic disorder, a moderate response rate in pivotal clinical trials and concerns over tolerability and weight gain can force clinicians to select alternative drug treatments, for which evidence-based support is sparse. BAART will assess predictors of efficacy, tolerability, and safety in 200 children five to seventeen years old with autistic disorder (AD) during a double-blind, randomized ten week treatment period with either risperidone or aripiprazole following a two-week single-blind placebo period. Responders who complete the study may continue with medication treatment for three months. Factors considered will include 1) psychiatric history; 2) symptom response; 3) psychosocial support; 4) measures of tolerability; 5) serum prolactin and brain-derived neurotrophic factor concentration; and 6) a variety of single nucleotide polymorphisms related to target genes for drug disposition and transport, response, and tolerability. Data analysis will allow assessment of gender, race, and multiple biomarkers for their predictive value on the clinical course and outcome to treatment with two widely used pharmacologic interventions for AD. The BAART project will result in evidence-based guidelines for selection and monitoring of drug treatment of children and adolescents with AD. $619,865.00 Treatments Question 4: Short-term Objective F Green dot: Objective has greater than or equal to the recommended funding. No Federal Medical University of South Carolina South Carolina New http://projectreporter.nih.gov/project_info_description.cfm?aid=7988490&icde=8914261 No URL available. No URL available. active 2010-2015
Project Id: 3790 2010-7 None Autism Research Institute Frye, Richard Survey on treatment for children with autism with and without seizures Seizures and epilepsy are common medical disorders that affect children with autism spectrum disorder. Individuals with ASD have a 3 to 22-fold increase in the risk of developing epilepsy as compared to the typical population. The relationship between epilepsy, seizures and ASD is complex, and there is a dearth of information regarding the treatment of individuals with ASD and seizures. This research will fund the implementation and analysis of a parent-friendly online survey for parents of children with ASD, both with and without seizures. The survey is comprehensive and includes questions on seizure characteristics, co-occurring medical conditions, changes in seizures that occur with seasons and allergy flares, and most importantly; the efficacy, effects on behavior and cognition, and side effects of traditional and non-traditional treatments. Local and national autism support groups will be contacted to ask them to advertise the survey and to assist with organization and analysis of the collected data. Responses will be broken down into subgroups in order to study the relationship between ASD and seizures in detail. $7,500.00 Treatments Question 4: Other Dot not applicable No Private Children's Learning Institute Texas New http://www.autism.com/index.php//about_2010_funded No URL available. No URL available. active 2010-2011
Project Id: 3943 AR093387 None Department of Defense Anagnostou, Evdokia Intranasal oxytocin for the treatment of children and adolescents with autism spectrum disorders (ASD) Autism or autism spectrum disorder (ASD) is characterized by social and communication deficits and repetitive behaviors/restricted interests. Typically, autistic disorder, Asperger syndrome, and PDD-NOS are included under the umbrella of ASD. When all forms of the syndrome are taken into consideration, ASD affects up to 1 in 150 individuals. There are currently no drugs approved for the treatment of social difficulties in autism. Oxytocin is a hormone produced in the brain. It is responsible for labor and breastfeeding and, for the longest time, it was considered to be a female hormone. However, scientists have recently reported that oxytocin in the brain is important in helping us understand others and their emotions, as well as relate to others. It may have an effect on our anxiety levels and on some repetitive behaviors. On an earlier small study, we showed that taking oxytocin as a spray through the nose twice a day helped adults with ASD understand people's emotions and reduced their repetitive behaviors, while being well tolerated.Based on the above, we believe it is appropriate that we start testing this medication in young individuals with autism. We propose to start with older children and adolescents, ages 12-17, who meet standard diagnostic criteria for autistic disorder or Asperger syndrome and have cognitive abilities within the typical range. The study will first recruit approximately 15 children and adolescents to make sure that the dose used in the adult study is still safe for these kids. Then 60 children and adolescents will receive either the oxytocin spray or a placebo spray for 12 weeks, while the investigators at University of Toronto/Bloorview Kids Rehab and University of Illinois at Chicago will monitor the effects of the spray, both good and bad. At the end, children who were given placebo can choose to try the oxytocin spray if they so wish.If we find oxytocin to be effective in our group, we will have more data to suggest that it or a similar compound may be of use to treat social difficulties in children and adolescents with autism. Because we will only recruit high functioning children ages 12-17, we can only generalize to children with similar age and cognitive levels. We do not anticipate any major side effects, given that breastfeeding women have been using this formulation for a long time. However, because we have not treated children and adolescents with this medication, we will be very vigilant in recording anything that could potentially be harmful, whether we think it is related or not to medication. By comparing the oxytocin group to the placebo group, we hope to be able to report on the safety of intranasal oxytocin in this group. We expect to be able to report clinically meaningful results by September of 2013. $0.00 Treatments Question 4: Short-term Objective C Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal Holland Bloorview Kids Rehabilitation Hospital Canada Ongoing http://cdmrp.army.mil/search.aspx No URL available. No URL available. active 2010-2013
Project Id: 3987 5R01MH079082-03 None National Institutes of Health Handen, Benjamin 1/3-Atomoxetine placebo and parent training in autism There have been relatively few randomized clinical trials of pharmacology for attention-deficit/hyperactivity (ADHD) in children with autism spectrum disorders (ASD). Atomoxetine (ATX) was approved by the FDA in January 2003 for treatment of ADHD, but only 4 studies of this drug have been conducted in children with ASD. Behavioral treatments, such as training parents in the use of behavioral interventions (PMT), are also standard treatments for ADHD in typically developing children. While PMT also has long been used in ASD, it has rarely been studied in combination with psychopharmacologic treatment in this population. This three-pronged 10-week trial in children with ASD will compare atomoxetine vs. placebo, PMT vs. no-PMT, and combined ATX/PMT vs. PMT alone. This trial has the potential to provide answers regarding optimal treatment (both psychosocial and pharmacological) for a sizeable subgroup of children with ASD who experience symptoms of over activity and inattention. $272,700.00 Treatments Question 4: Short-term Objective F Green dot: Objective has greater than or equal to the recommended funding. No Federal University of Pittsburgh Pennsylvania Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=7800329&icde=6645080 No URL available. No URL available. active 2008-2013
Project Id: 3841 5M01RR000188-46 6565 National Institutes of Health Hopkins, Bobbi Treatment of sleep problems in children with autism spectrum disorder with melatonin: A double-blind, placebo-controlled study Children with autism spectrum disorder (ASD) experience high rates of sleep disturbances that potentially contribute to the severity of their cognitive and behavioral dysfunction and to poor quality of life for themselves and their families. This research will examine whether sleep problems in children with ASD are related to alterations in the secretion of melatonin (MT), a hormone that plays an important role in regulating the circadian cycle and sleep. It will also determine the efficacy of MT for improving sleep in children with ASD. Participants in the study will be recruited from the Autism Treatment Network (ATN) and parents will complete a validated sleep questionnaire to assess presence or absence of sleep problems in their child. Levels of melatonin will be measured before and after a child's usual bedtime and the total 24-hour MT production will be determine via urine samples from each child. In addition, a double-blind, randomized trial of three oral doses of MT (3, 6, 9 mg) and placebo in children ages 4-9 years will establish efficacy of its use in managing sleep problems in children with ASD. Data from this study will provide important information concerning circadian rhythm dysregulation in ASD and will support the development of future studies using melatonin to modify and correct abnormal circadian rhythms. $8,775.00 Treatments Question 4: Short-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal Baylor College of Medicine Texas Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=8166701&icde=8965742 No URL available. No URL available. active 2009-2010
Project Id: 3838 2003 None Autism Speaks Hopkins, Bobbi Treatment of sleep problems in children with autism spectrum disorder with melatonin: A double-blind, placebo-controlled study This study will examine whether sleep problems in children with autism spectrum disorder (ASD) are related to alterations in the production of melatonin (MT), a hormone that plays an important role in regulating the sleep-wake cycle. This study will also examine whether MT is effective in improving sleep in children with ASD. The two hypotheses that will be tested are: 1) Children with ASD and sleep problems will have a delayed sleep-wake cycle and/or decreased MT production compared with those without sleep problems; and 2) treatment with MT will be associated with improved sleep and behavior. Sleep disturbance will be measured using a standardized questionnaire. The MT levels will be measured in saliva in two groups of children with ASD both with and without sleep problems. Total 24-hour MT production will be determined from urine samples in these same two groups. Eligible participants will then be enrolled in a trial of three oral doses of MT (3, 6, 9 mg) and a placebo. $127,500.00 Treatments Question 4: Short-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Private Baylor College of Medicine Texas Ongoing http://www.autismspeaks.org/science/grants/treatment-sleep-problems-children-autism-spectrum-disorder-melatonin-double-blind-pla?destination=science%2Fgrant-search%2Fresults%2FTreatment+of+sleep+problems+in+children+with+autism+spectrum+disorder+with+melatonin%3A+A+double-blind%2C+placebo-controlled+study No URL available. No URL available. active 2007-2011
Project Id: 3740 AS073084 None Department of Defense Johnson, William Developing treatment, treatment validation, and treatment scope in the setting of an autism clinical trial This proposal has three linked and interacting projects designed to (1) develop new therapy for children with autism, (2) develop means of validating and monitoring responses to therapy assessed through clinical changes in the children and the amount of certain lipid biomarkers that they excrete in their urine, and (3) develop means for selecting children with autism likely to respond to this therapy based upon the children's genotypes, that is, the pattern of variants of certain key genes that the children have.The rationale is that growing evidence supports oxidative stress as a mechanism that contributes to autism. A key omega-3 fatty acid called docosahexaenoic acid (DHA) is a normal substance that is present in large amounts in the brain and that has properties that oppose oxidative stress. Therapeutic trials of agents containing DHA appear promising in autism. We were first to find that urinary excretion of a lipid biomarker of oxidative stress is increased in autism. In a preliminary study, we found that the amount of this biomarker excreted in children with autism correlated with a certain genotype in the children.We propose to carry out a rigorous therapeutic trial to assess the effect of DHA vs. placebo treatment on the global severity, behavioral symptoms, and functional ability of child and adolescent autistic disorder, in a 12-week double blind placebo-controlled parallel study (Project 1). We propose to measure changes in urinary excretion of this biomarker, isoprostane, during DHA therapy in the children with autism and develop new urinary biomarkers that may be even more informative (Project 2). We also propose to determine genotypes of the children undergoing DHA therapy since we found one gene variant, GSTM1*0, that correlated with the amount of isoprostane that children with autism excreted in their urine in a preliminary study (Project 3). We will test whether this gene variant and new gene variants that we will study will correlate also with response to DHA treatment (Project 3). We hope that this will allow us to predict which children with autism will respond to DHA therapy. This may also give us insight into whether some children with autism have a gene variant that interferes with DHA metabolism.This will help all children with autism by developing a new method of therapy, by developing a method to monitor the success of the therapy and show that it is working and by developing a method to determine which children with autism will respond best to the therapy.The clinical benefits will be assessed for global severity of autism, and for behavioral and functional aspects of autism. The therapy is safe and has no known risks.What is the projected time to achieve a consumer-related outcome? The study is a direct therapeutic trial. If the trial is successful, this therapy will be available to consumers by the end of the three-year project.The likely contributions of this study are (1) development of new therapy for autism, (2) development of a way to determine if the therapy is working in a child with autism and how well it is working, and (3) to develop a way of determining which children will respond best to the therapy. $0.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal University of Medicine & Dentistry of New Jersey - Robert Wood Johnson Medical School New Jersey Ongoing http://cdmrp.army.mil/search.aspx No URL available. No URL available. active 2008-2012
Project Id: 3752 1R01MH083747-01A2 None National Institutes of Health McCracken, James 3/4-RUPP autism network: Guanfacine for the treatment of hyperactivity in PDD This is a multi-site collaborative research project from The Research Units on Pediatric Psychopharmacology [RUPP] Autism Network (Indiana University, Seattle Children's Research Institute, UCLA, and Yale University). Recent data indicate that as many as 50% of children with pervasive developmental disorders (PDDs) have moderate to severe problems of hyperactivity and impulsiveness. Consensus is lacking on how to treat children with PDD accompanied by hyperactivity. Compared to typically developing children with ADHD, children with PDD often show less benefit and greater side effect burden. Guanfacine is commonly used in this population, but poorly studied. Pilot data indicate that guanfacine is a promising treatment for hyperactivity in children with PDD with a good tolerability profile. In addition, biomarkers have been identified (genetic and neurochemical) that may be associated with positive effects. This study involves an 8-week randomized, double-blind, placebo- controlled trial of guanfacine for 170 children (ages 5-13 years) with PDD accompanied by hyperactivity and impulsiveness. Subjects who show a positive response will be invited to enter an 8-week Extension phase (treatment mask will not be broken). The treatment blind will be broken for children who do not achieve a positive response in the double-blind phase. Children who show no change or deterioration on placebo will be treated with guanfacine in an 8-week Open-label phase. Children who show a partial response to guanfacine will be randomly assigned to a 4-week add-on trial of methylphenidate or placebo to evaluate the potential benefits of combined treatment. Fifty subjects are expected to enter this pilot trial. The role of gene variants and urinary adrenergic/noradrenergic measures as biomarkers in moderating response to guanfacine on primary efficacy measures and adverse effects will also be explored. $391,103.00 Treatments Question 4: Long-term Objective C Green dot: Objective has greater than or equal to the recommended funding. No Federal University of California, Los Angeles California New http://projectreporter.nih.gov/project_info_description.cfm?aid=7890096&icde=8916110 No URL available. No URL available. active 2010-2013
Project Id: 3744 5P50HD055784-04 5 National Institutes of Health McCracken, James ACE Center: Understanding repetitive behavior in autism This grant provides support for an NIH Autism Center of Excellence (ACE). Repetitive behaviors form a core feature of autism spectrum disorders (ASD), but the understanding of the neural basis of stereotypy and repetitive behaviors in ASD and their treatment is limited. Recently, risperidone, an antipsychotic, was observed to robustly reduce stereotypic behaviors in a sample of children and adolescents with autism. The proposed study attempts to increase understanding of repetitive behaviors in ASD and its treatment by examining the changes in key neural circuits associated with risperidone treatment using functional magnetic resonance imaging (fMRI). The proposed clinical trial and study design provide a unique opportunity to study the biology of treatment response on the target of repetitive behaviors which could cast a broader light on the nature of brain dysfunction across these similar parallel distributed brain systems and their interactions. $326,665.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal University of California, Los Angeles California Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=8129724&icde=8970213 No URL available. No URL available. active 2007-2012
Project Id: 3753 1R01MH083739-01A2 None National Institutes of Health McDougle, Christopher 1/4-RUPP autism network: Guanfacine for the treatment of hyperactivity in PDD This is a multi-site collaborative research project from The Research Units on Pediatric Psychopharmacology [RUPP] Autism Network (Indiana University, Seattle Children's Research Institute, UCLA, and Yale University). Recent data indicate that as many as 50% of children with pervasive developmental disorders (PDDs) have moderate to severe problems of hyperactivity and impulsiveness. Consensus is lacking on how to treat children with PDD accompanied by hyperactivity. Compared to typically developing children with ADHD, children with PDD often show less benefit and greater side effect burden. Guanfacine is commonly used in this population, but poorly studied. Pilot data indicate that guanfacine is a promising treatment for hyperactivity in children with PDD with a good tolerability profile. In addition, biomarkers have been identified (genetic and neurochemical) that may be associated with positive effects. This study involves an 8-week randomized, double-blind, placebo- controlled trial of guanfacine for 170 children (ages 5-13 years) with PDD accompanied by hyperactivity and impulsiveness. Subjects who show a positive response will be invited to enter an 8-week Extension phase (treatment mask will not be broken). The treatment blind will be broken for children who do not achieve a positive response in the double-blind phase. Children who show no change or deterioration on placebo will be treated with guanfacine in an 8-week Open-label phase. Children who show a partial response to guanfacine will be randomly assigned to a 4-week add-on trial of methylphenidate or placebo to evaluate the potential benefits of combined treatment. Fifty subjects are expected to enter this pilot trial. The role of gene variants and urinary adrenergic/noradrenergic measures as biomarkers in moderating response to guanfacine on primary efficacy measures and adverse effects will also be explored. $366,035.00 Treatments Question 4: Long-term Objective C Green dot: Objective has greater than or equal to the recommended funding. No Federal Indiana University-Purdue University Indianapolis Indiana New http://projectreporter.nih.gov/project_info_description.cfm?aid=7890695&icde=0 No URL available. No URL available. active 2010-2013
Project Id: 3754 5R01MH077600-04 None National Institutes of Health McDougle, Christopher Targeted pharmacologic interventions for autism This project will examine the efficacy and tolerability of atomoxetine for treatment of hyperactivity associated with autism in children and adolescents. An 8-week randomized clinical trial will be conducted, and patients that respond well to short-term treatment with atomoxetine will be followed for an additional 10 months. Genetic polymorphisms of the cytochrome P450 2D6 gene will also be analyzed to determine their relationship to response and tolerability to atomoxetine. This research addresses the need to find safer and more effective medical treatments for autism and is also unique in its plan to identify genetic predictors of response to drug treatment in autism. $370,481.00 Treatments Question 4: Long-term Objective C Green dot: Objective has greater than or equal to the recommended funding. No Federal Indiana University-Purdue University Indianapolis Indiana Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=7799941&icde=6669587 No URL available. No URL available. active 2007-2012
Project Id: 3750 5717 None Autism Speaks McDougle, Christopher Mirtazapine treatment of anxiety in children and adolescents with pervasive developmental disorders Pervasive developmental disorders (PDDs) are life-long childhood neurobiological disorders that cause marked problems with social interaction and communication. In addition to the core impairments in socialization and language, many persons with PDDs often experience high levels of anxiety. Little to no research has been done on treating anxiety symptoms in ASDs, yet preliminary studies suggest it is a common and disabling symptom in many persons. Currently used medications are frequently ineffective or cause troublesome adverse effects. The primary objective of this application is to conduct a preliminary placebo controlled trial of mirtazapine for the treatment of anxiety associated with PDDs. Mirtazapine is novel in that it has both noradrenergic and serotonergic properties. The central hypothesis of this project is that mirtazapine will improve anxiety when administered to children and adolescents with PDD. Thirty children and adolescents (ages 5-17 years) will be randomized to either mirtazapine (n=20) or placebo (n=10) and assessed over 10 weeks of treatment. Finding preliminary evidence of mirtazapine efficacy will be highly significant given the paucity of research involving anxiety in PDD. In addition to improving our understanding of anxiety and its treatment in PDD, results from this trial would be crucial in the development of a larger multi-site clinical trial. When completed, this study will be the first prospective trial of mirtazapine in ASDs, as well as one of the first to examine treatments for anxiety in ASDs. Furthermore, it will give important information about the utility of various outcome measures in measuring anxiety in ASDs. $99,974.00 Treatments Question 4: Long-term Objective C Green dot: Objective has greater than or equal to the recommended funding. No Private Indiana University Indiana New http://www.autismspeaks.org/science/grants/mirtazapine-treatment-anxiety-children-and-adolescents-pervasive-developmental-disord?destination=science%2Fgrant-search%2Fresults%2FMcDougle%2C+Christopher No URL available. No URL available. active 2010-2013
Project Id: 3741 AS073084P2 None Department of Defense Novotny, Sherie Developing treatment, treatment validation, and treatment scope in the setting of an autism clinical trial This proposal has three linked and interacting projects designed to (1) develop new therapy for children with autism, (2) develop means of validating and monitoring responses to therapy assessed through clinical changes in the children and the amount of certain lipid biomarkers that they excrete in their urine, and (3) develop means for selecting children with autism likely to respond to this therapy based upon the children's genotypes, that is, the pattern of variants of certain key genes that the children have.The rationale is that growing evidence supports oxidative stress as a mechanism that contributes to autism. A key omega-3 fatty acid called docosahexaenoic acid (DHA) is a normal substance that is present in large amounts in the brain and that has properties that oppose oxidative stress. Therapeutic trials of agents containing DHA appear promising in autism. We were first to find that urinary excretion of a lipid biomarker of oxidative stress is increased in autism. In a preliminary study, we found that the amount of this biomarker excreted in children with autism correlated with a certain genotype in the children.We propose to carry out a rigorous therapeutic trial to assess the effect of DHA vs. placebo treatment on the global severity, behavioral symptoms, and functional ability of child and adolescent autistic disorder, in a 12-week double blind placebo-controlled parallel study (Project 1). We propose to measure changes in urinary excretion of this biomarker, isoprostane, during DHA therapy in the children with autism and develop new urinary biomarkers that may be even more informative (Project 2). We also propose to determine genotypes of the children undergoing DHA therapy since we found one gene variant, GSTM1*0, that correlated with the amount of isoprostane that children with autism excreted in their urine in a preliminary study (Project 3). We will test whether this gene variant and new gene variants that we will study will correlate also with response to DHA treatment (Project 3). We hope that this will allow us to predict which children with autism will respond to DHA therapy. This may also give us insight into whether some children with autism have a gene variant that interferes with DHA metabolism.This will help all children with autism by developing a new method of therapy, by developing a method to monitor the success of the therapy and show that it is working and by developing a method to determine which children with autism will respond best to the therapy.The clinical benefits will be assessed for global severity of autism, and for behavioral and functional aspects of autism. The therapy is safe and has no known risks.What is the projected time to achieve a consumer-related outcome? The study is a direct therapeutic trial. If the trial is successful, this therapy will be available to consumers by the end of the three-year project.The likely contributions of this study are (1) development of new therapy for autism, (2) development of a way to determine if the therapy is working in a child with autism and how well it is working, and (3) to develop a way of determining which children will respond best to the therapy. $0.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal University of Medicine & Dentistry of New Jersey - Robert Wood Johnson Medical School New Jersey Ongoing http://cdmrp.army.mil/search.aspx No URL available. No URL available. active 2008-2012
Project Id: 3745 5P50HD055751-04 3 National Institutes of Health Owley, Thomas ACE Center: The pharmacogenetics of treatment for insistence sameness in autism This grant provides support for an NIH Autism Center of Excellence (ACE). Insistence on Sameness (IS) is a common feature of ASD, and IS has been hypothesized to be due to improper serotonin regulation in this population. Many patients with ASD and IS benefit from treatment with medications that affect serotonin, in particular the selective serotonin reuptake inhibitors (SSRIs); however, there is little information available about which particular IS symptoms respond to SSRIs or which patients might be better candidates based on the presenting symptoms or their genetic variation. Clinical response to selective serotonin reuptake inhibitors (escitalopram) and effect of genetic variation in the serotonin transporter/receptor genes (SLC6A4 and HTR2A) on Insistence on Sameness (IS) symptoms will be explored in this pharmacogenetic study. $382,540.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal University of Illinois at Chicago Illinois Ongoing http://projectreporter.nih.gov/project_info_description.cfm?aid=8129548&icde=8970134 No URL available. No URL available. active 2007-2012
Project Id: 3840 UA3 MC 11054 None Health Resources and Services Administration Perrin, James Autism Intervention Research Network on Physical Health (AIR-P network) This multi-site research network conducts research on evidence-based practices for interventions to improve the physical health and well-being of children and adolescents with autism spectrum disorders (ASD) and other developmental disabilities. The network also develops guidelines and validates tools for interventions and disseminates network findings broadly to health professionals and the public, especiallly families impacted by ASD and other developmental disabilities. $3,651,425.00 Treatments Question 4: Short-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Federal Massachusetts General Hospital Massachusetts Ongoing http://www.mchb.hrsa.gov/research/project_info.asp?ID=106 No URL available. No URL available. active 2009-2011
Project Id: 3737 5907 None Autism Speaks Reiss, Allan Association of cholinergic system dysfunction with autistic behavior in fragile X syndrome: Pharmacologic and imaging probes The term "autism" is being increasingly recognized as a diagnostic category that covers a large number of, as yet, unidentified brain disorders of childhood onset. In contrast, Fragile X syndrome (FraX) is a common, readily identified genetic condition associated with greatly increased risk for autistic behavior. Thus, FraX offers a unique opportunity to study how genetic and environmental factors lead to autistic behavior, and how disease-specific treatments can reduce such behaviors. Preliminary studies from the research team and others indicate that dysfunction of a specific neurochemical system of the brain (the "cholinergic" system) is relevant to FraX and that intervention to augment this system may have beneficial effects for both behavior and learning. Two objectives are proposed for this research. First, to conduct a controlled clinical trial to assess whether a medicine that increases cholinergic function (donepezil) can reduce autistic behavior and improve cognition in persons with FraX; and second, to use special magnetic resonance imaging (MRI) techniques to assess change in brain function and neurochemistry associated with donepezil treatment. Autistic behaviors occurring in FraX are similar to those observed in idiopathic (non-FraX) autism. Like idiopathic autism, behavioral and learning problems in FraX contribute to impairments in social development, disturbance in the parent-child relationship and educational failure. Accordingly, the study of FraX as a "comprehensible" model for autism has been increasingly proposed. As hypotheses about cholinergic dysfunction have been offered for idiopathic autism as well as FraX, the results of the study hold promise for informing future research in autistic populations who share genetic or neurobiological links to FraX $94,832.00 Treatments Question 4: Long-term Objective A Yellow dot: Objective has some degree of funding, but less than the recommended amount. No Private Stanford University California New http://www.autismspeaks.org/science/grants/association-cholinergic-system-dysfunction-autistic-behavior-fragile-x-syndrome-pharm?destination=science%2Fgrant-search%2Fresults%2Freiss No URL available. No URL available. active 2010-2013
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